European Commission decides not to renew the market authorisation of Translarna (ataluren)
The European Commission has announced that it will not renew the market authorisation of Translarna (ataluren). This follows the European Medicine Agency (EMA) finalising its review of Translarna in December. It concluded that the medicine’s effectiveness for treating Duchenne muscular dystrophy (DMD) could not be confirmed. This decision does not impact Translarna’s approval in the UK.
Conditional approval
Translarna was first approved for DMD by the EMA in 2014. Because there was uncertainty over the efficacy of the medicine at the time, it was given a conditional marketing authorisation.
The company that manufactured it – PTC Therapeutics, Inc. – was asked to do more research to demonstrate Translarna’s benefits.
Review of Translarna data
The EMA reviewed the available information, and reached an initial opinion to not renew the conditional marketing authorisation in 2023. It gave this recommendation to the European Commission, who could then make a legally binding decision applicable to all European Union member states. PTC appealed the recommendation, and the EC asked the EMA to think again.
Since then, the EMA has revisited the data and looked at new studies, but maintained its negative opinion in January 2024, June 2024, October 2024, and at the end of March 2025.
Impact in UK
Translarna remains approved for use in the NHS. While Translarna’s authorisation was granted in 2014 when the UK was still a member of the EU, the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has overseen giving medicines licencing since 2021.
We are monitoring the status of Translarna’s approval in the UK, and we will share any information if there is an update.